Pilot Study of Reduced Venetoclax Exposure

NCT07163793 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 24-0579
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

Brief Summary

Pilot Study of Reduced Venetoclax Exposure

Conditions

Acute Myeloid Leukemia in Remission

Keywords

AML

Outcome Measures

Primary Outcomes (1)

• Venetoclax Tolerability Rate

  Tolerability rate defined as the proportion of patients who do not experience grade 4 neutropenia (per the CTCAE v5.0) for over 7 continuous days within the first three cycles of maintenance therapy (90 days). Patient will be evaluated on Day 1 of each cycle. Laboratory studies of prior cycle will be reviewed on that day to identify if patient had \>7 continuous days of cytopenia.

  12 months

Secondary Outcomes (10)

• Evaluation of Event-Free Survival (EFS)

  12 months

• Time to Detriment

  48 months

• Number of pRBC transfusion

  48 months

• Evaluate Number of Hospital Admissions

  12 months

• Rate of adverse events/toxicities

  48 months

Study Arms (2)

Azacytadine + Venetoclax

ACTIVE COMPARATOR

Drug: Azacitidine; Drug: Venetoclax

Decitabine + Venetoclax

ACTIVE COMPARATOR

Drug: Decitabine; Drug: Venetoclax

Interventions

Azacitidine DRUG

Given Day 1-7 with Venetoclax Day 1-14 every 28 days until off study

Also known as: Vidaza

Azacytadine + Venetoclax

Decitabine DRUG

Given Day 1-5 with Venetoclax Day 1-14 every 28 days until off

Also known as: Dacogen

Decitabine + Venetoclax

Venetoclax DRUG

Venetoclax up to 400mg a day on Day 1-14 every 28 days until off in combination with Azacitidine or Decitabine

Also known as: Venclexta, Venclyxto

Azacytadine + Venetoclax; Decitabine + Venetoclax

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Stated willingness to comply with all study procedures and availability for the duration of the study
• Ability to take oral medication and be willing to adhere to the study regimen
• Diagnosed by current WHO or ICC criteria with Acute Myeloid Leukemia and treated for initial induction therapy with one of two regimens:
• Azacitidine administered subcutaneously at a dose of 75mg/m2/day X 7 days in combination with VEN (21-28 days/cycle)
• Decitabine administered intravenously at a dose of 20mg/m2/day administered in combination with VEN (21-28 days/cycle)
• Achieving morphological CR/CRi by bone marrow biopsy with \<5% blasts within 3 cycles. See Appendix 2 for definitions.
• Consent to be obtained within 10 days (+/- 3 days) of bone marrow biopsy report showing morphological remission. C1D1 of trial to be initiated within 10 days (+/- 7 days) of bone marrow biopsy report showing morphological remission.
• ECOG 0-3
• Intensive treatment ineligible; transplant ineligible or refusal of transplant
• Patient must be able to understand and sign informed consent and additional study documents
• On C1D1 of trial, patient must have count recovery with ANC \>1000, platelets \> 50, Hemoglobin \> 7.7 and without transfusion support for 7 days.
• No growth factor (G-CSF) use in 14 days prior to C1 D1 of trial.

You may not qualify if:

• Treatment with another investigational drug
• Use of growth factor (G-CSF) within the last 14 days prior to C1D1 of trial treatment.
• On concomitant targeted therapy such as FLT3 inhibitor or IDH1/2 inhibitor.
• Subject has received treatment prior to induction with the following:
• i. Prior hypomethylating agent or BCL-2 inhibitor for either AML or MDS other than for induction prior to enrollment.
• ii. Prior CAR-T cell therapy. iii. Experimental or investigational drug therapy for 14 days prior to study entry leukemia-directed therapies.
• Subject has:
• i. Acute promyelocytic leukemia (APL) with t(15;17). ii. Presence of t(9;22) given the potential indication for concurrent tyrosine kinase therapy.
• iii. Known active CNS involvement with AML.

Sponsors & Collaborators

• Northwell Health: lead

Study Sites (1)

Zuckerberg Cancer Center

New Hyde Park, New York, 11042, United States

RECRUITING

MeSH Terms

Interventions

Azacitidine; Decitabine; venetoclax

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Boiclair Stephanie, MD

  Northwell Health

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Heme Referral Team

CONTACT

(516) 734-8896; ci-hematology@northwell.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: September 9, 2025
• Study Start: October 7, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2028
• Last Updated: November 20, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)